269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy – What have we learned? 9–11 December 2022, Hoofddorp, The Netherlands

Naarding, Karin J.; Stimpson, Georgia; Ward, Susan J.; Goemans, Nathalie; McDonald, Craig; Mercuri, Eugenio; Muntoni, Francesco; Goemans, Nathalie; McDonald, Craig; Mercuri, Eugenio; Muntoni , Francesco; Ward, Susan J.; Bettica, Paolo; Binks, Michael; Duong, Tina; Ferreiro, Ana; Furlong, Patricia; Guglieri, Michela; Hendrix, Suzanne; Henricson, Erik; Hogrel, Jean-Yves; Klein, Matthew; Leffler, Mindy; Martinez, Terina; Naarding, Karin J.; Niks, Erik; Servais, Laurent; Signorovitch, James; Stimpson , Georgia; Van Ieperen, Frank; Vroom, Elizabeth

doi:10.1016/j.nmd.2023.10.003

Neuromuscular Disorders

2023

DOI: 10.1016/j.nmd.2023.10.003

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269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy – What have we learned? 9–11 December 2022, Hoofddorp, The Netherlands

Karin J. Naarding,

Georgia Stimpson,

Susan J. Ward

et al.

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New Horizons of Model Informed Drug Development in Rare Diseases Drug Development

Mitra,

Tania,

Ahmed

et al. 2024

Clin Pharma and Therapeutics

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Model‐informed approaches provide a quantitative framework to integrate all available nonclinical and clinical data, thus furnishing a totality of evidence approach to drug development and regulatory evaluation. Maximizing the use of all available data and information about the drug enables a more robust characterization of the risk–benefit profile and reduces uncertainty in both technical and regulatory success. This offers the potential to transform rare diseases drug development, where conducting large well‐controlled clinical trials is impractical and/or unethical due to a small patient population, a significant portion of which could be children. Additionally, the totality of evidence generated by model‐informed approaches can provide confirmatory evidence for regulatory approval without the need for additional clinical data. In the article, applications of novel quantitative approaches such as quantitative systems pharmacology, disease progression modeling, artificial intelligence, machine learning, modeling of real‐world data using model‐based meta‐analysis and strategies such as external control and patient‐reported outcomes as well as clinical trial simulations to optimize trials and sample collection are discussed. Specific case studies of these modeling approaches in rare diseases are provided to showcase applications in drug development and regulatory review. Finally, perspectives are shared on the future state of these modeling approaches in rare diseases drug development along with challenges and opportunities for incorporating such tools in the rational development of drug products.

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New Horizons of Model Informed Drug Development in Rare Diseases Drug Development

Mitra,

Tania,

Ahmed

et al. 2024

Clin Pharma and Therapeutics

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show abstract

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269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy – What have we learned? 9–11 December 2022, Hoofddorp, The Netherlands

Cited by 1 publication

References 70 publications

New Horizons of Model Informed Drug Development in Rare Diseases Drug Development

New Horizons of Model Informed Drug Development in Rare Diseases Drug Development

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